Division of Hematology

1. Division of Hematology Basil Golding M.D. Division Director Site Visit 2005 Laboratories of Plasma Derivatives, and Hemostasis

2. Division of Hematology Research/Review Units LBVB LCH LH LPD CRB Biochemistry & Vascular Biology Cellular Hematology Hemostasis Plasma Derivatives Clinical Review Branch PIs: 3 2 2 4 Total = 11

3. CBER Mission MISSION: To ensure the safety, purity, potency and effectiveness of biological products including vaccines, blood and blood products, and cells, tissues and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions or injury. Through our mission, we also help to defend the public against the threats of emerging infectious diseases and bioterrorism.

4. Division of Hematology Scope of Regulation and Research • Research helps solve regulatory problems (Critical Path) and serves to enhance the expertise of scientific investigators who have review responsibility for these products • Scientific evaluation of biologic products derived from blood include those • isolated from blood or plasma • analogous materials derived by recombinant DNA technology • Clinical applications include : • prophylaxis and/or treatment of bleeding disorders • shock • infectious diseases • immunological deficits • replacement therapy in congenital or acquired deficiencies.

5. Division of Hematology: Regulatory Products and Process • Applications and products: • biologics, drugs, and devices • Diverse complex proteins and carbohydrate polymers • Decisions - based on scientific data showing safety, efficacy and purity • decision-making process • internal review • presentations to Advisory Committees • conferences with manufacturers • workshops.

6. Division of Hematology:Regulatory Process • Evaluation process of products • scientific review • laboratory investigation if needed • lot release tests • standards are developed for lot release of different products and are often distributed as CBER and/or international standards • Surveillance • Inspections • Adverse Event Reports

7. Division of Hematology:Other Tasks • Policy and Guidance documents • Harmonization • Liaisons: Government Agencies (e.g. NIH, CDC) Regulatory agencies of foreign governments International bodies Consumer groups Non-profit organizations Industry • Communications • Websistes • Letters

8. Division of HematologyResearch • Research Topics • coagulation • immunology • protein structure and function • blood-borne viruses and TSE/prions • oxygen-carrying compounds • platelet structure and function

9. Laboratory of Plasma Derivatives (LPD) • Review responsibility: immune globulin products • Scientific Program: • immune system • contaminants of IG: plasma proteins, and microorganisms • Staff (n =16, including 1 regulatory scientsist) • Dr. Scott’s (Lab Chief) section: 1 Staff Fellow, 1 Visiting Associate, and 2 Biologists (n = 5). • Dr. Golding’s section consists of three staff fellows, and one staff scientist (n = 5). • Dr. Yu’s section consists of 1 Visiting Scientist, 2 Visiting associates, and 1 ORISE fellow (n = 5) 

10. Lab of Hemostasis • Review Responsibility • regulation of coagulation products, both plasma-derived and recombinant proteins. • Staff (n = 10, including 5 regulatory scientists) • Dr. Lozier’s section consists of one Staff Fellow and one Biologist (n = 3) • Dr. Kimchi-Sarfaty’s section consists of 1 IRTA (n = 2)

11. Purpose of Site Visit • Review the research programs: • Laboratory of Plasma Derivatives (LPD) • Laboratory of Hemostasis (LH) • In-Depth Review of the following P.I.s Programs: • Drs. Golding, Scott, Yu, and Lozier • Decisisons/Advice • Promotion of Dr. Scott from GS-14 to GS-15 • Conversion of Dr. Lozier from a Senior Staff Felow to a permanent position, GS-14 • Drs. Pei Zhang, a Visiting Scientist, and Charles Wu, Visiting Associate will have Progress Reviews.